Pompe disease

The efficacy of avalglucosidase alfa compared to alglucosidase alfa as measured by the EuroQol 5-dimension 5-level (EQ-5D-5L) from the phase 3 COMET trial in late-onset Pompe disease (LOPD) is presented. EQ-5D-5L, a multi-attribute utility measure of health-related quality of life, was collected in treatment-na ïve LOPD patients. Change from baseline to week 49 in the five dimensions of EQ-5D-5L, including mobility, usual activities, self-care, pain/discomfort and anxiety/depression; the derived patient's health state/index (range: 0 to 1); and the visual analogue scale (VAS, self-rated health on a 0 [wor st health] to 100 [best health]) were compared for the two treatment groups using Wilcoxon-Mann-Whitney test at the nominal level.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research